STUTTGART, Germany I June 23, 2017 I Overlooked for many years, RNA languished as the ugly stepchild of nucleic acid vaccine development and was thought to be a poor choice for a therapeutic agent given its short half-life in vivo and its immunogenicity. But two of the main problems of messenger RNA (mRNA) technologies, i.e. stability and manufacturing, sufficiently have been solved and optimized nanoparticle carrer technologies enable targeted mRNA delivery in vivo.
The technological progress has made pilot projects feasible for biopharmaceutical and major pharma companies. Upfront payments of some of the collaboration agreements between key mRNA players and Big Pharma reached US$ 240 mln and US$ 310 mln, respectively. Some of the deals between mRNA companies and rare disease specialist biopharmaceutical companies have a potential value of up to US$ 2.4 bln and US$ 3.2 bln, respectively, if all milestones will be met. Over the last years, companies with mRNA technologies have attracted mroe than US$ 3.4 bln from equity investments and partnership payments. In addition, funding from governmental institutions and philanthropic organizations has been been crucial for development of mRNA technologies (vaccines and antibodies) for infectious diseases.
mRNA is a rather versatile technology and offers a number of advantages. mRNA lacks genomic integration and its use results in transient expression of the encoded protein. This favorable safety profile makes mRNA especially attractive for vaccines and gene editing. mRNA is well defined chemically which ensures reproducible manufacturing at high yield, purity and activity. Improvements of lipid nanoparticle formulations as a vehicle for in vivo systemic delivery of mRNA has greatly favored the development of in vivo transfection strategies.
Scope of clinical mRNA applications
|Cancer Vaccines||Infectious Disease Vaccines||In vivo Therapeutics||Gene Editing|
|– Standardized preselected
– Individualized neoantigens
|– Prophylactic vaccines
– Therapeutic vaccines
– Synthetic self-amplifying mRNA vaccines for pandemic outbreaks
|– Therapeutic proteins
– Therapeutic antibodies
|– Ex vivo gene editing of gene defects
– In vivo gene editing of gene defects
– Ex vivo gene editing of autologous and allogeneic T-cells
In a new report released by La Merie Publishing, the competitive landscape of mRNA Vaccines & Therapeutics is described and analyzed for pipelines, technologies, stakeholders, financing and deals.
This report „mRNA Vaccines & Therapeutics 2017: an industry analysis of technologies, pipelines, stakeholders and deals“ as of June 2017 brings you up-to-date regarding key players, key technologies, profiles of key mRNA vaccines and therapeutics in development, business deals and private and public financing rounds. The report analyzes the mRNA pipeline and stakeholders in the field. The report highlights the value of mRNA product candidates in terms of partnering terms and conditions, venture and private financing and non-dilutive funding.
About La Merie
La Merie Publishing is a Business Intelligence enterprise fully dedicated to provide high quality R&D information to the biopharmaceutical industry. La Merie offers individual consultancy services and publishes reports and periodicals. For more information visit www.lamerie.com and www.PipelineReview.com, the Biologics News Center and Online Store of La Merie Publishing.
SOURCE: La Merie Publishing